United Therapeutics (UTHR) investor relations material

Key strategic focus areas

• Emphasis on IPF (idiopathic pulmonary fibrosis) and super-prostacyclin (ralinepag) as main growth drivers, expected to transform the business over the next several years.

• IPF represents a large, underserved market with 100,000 patients in the U.S. and high unmet need due to limited efficacy of current therapies.

• Super-prostacyclin (ralinepag) is positioned as a next-generation, once-daily therapy with superior pharmacokinetics and pharmacodynamics, aiming to become the leading treatment for pulmonary hypertension.

• Recent TETON 2 trial data for Tyvaso in IPF showed dramatic improvement in forced vital capacity, described as a game changer by key opinion leaders.

• Plans to file for regulatory approval of Tyvaso in IPF this summer, with FDA approval targeted for next year.

Clinical development and innovation

• TETON 1 confirmatory trial for Tyvaso in IPF is fully enrolled and will be unblinded in the first half of the year; expectation is for results to mirror TETON 2.

• Open-label extension data for ralinepag shows sustained improvement in exercise capacity, supporting its potential as the most prescribed pulmonary hypertension drug.

• Use of advanced computational biology (CLIME model) to simulate clinical trials, achieving results closely matching in vivo outcomes and accelerating development timelines.

• Drug-device combination products, such as Tyvaso DPI, are a core competency, enabling deeper lung delivery and improved patient outcomes.

• Proprietary next-generation inhalers are in development, with internal manufacturing capabilities being established.

Business development and pipeline expansion

• Orphan oncology, particularly pediatric neuroblastoma (Unituxin), is a key area for new business development, with plans to expand into other rare cancers.

• Xenotransplantation platform for kidneys and hearts is advancing, with two FDA-approved clinical trials and modular facilities capable of producing over 1,000 organs per year.

• IND submissions for additional xenotransplant studies (xeno-heart, xeno-thymic heart) are expected in the first half of the year.

• Development of personalized, cellularized lungs using iPSC or HLA-matched cells is underway, targeting regulatory approval by decade's end.

• Quality assurance and control are prioritized in scaling xenotransplantation, with modular facility expansion to ensure safety and compliance.